pure red-cell aplasia
pure red-cell aplasia
Explore linked clinical trials. Standard reference: MONDO:000170514 results · Page 1 of 1
Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| Cancer in Inherited Bone Marrow Failure Syndromes | Bone Marrow Failure Disorders, Congenital Abnormalities, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DNA repair disease, Diamond-Blackfan anemia, Digestive System Diseases, Fanconi anemia, Hemic and Lymphatic Diseases, Infant, Newborn, Diseases, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Nutritional and Metabolic Diseases, Shwachman-Diamond syndrome, Skin Abnormalities, Skin and Connective Tissue Diseases, X-linked disease, anemia, aplastic anemia, bone marrow disorder, dyskeratosis congenita, exocrine pancreatic insufficiency, hematologic disorder, hereditary disease, hereditary skin disorder, inherited aplastic anemia, lipomatosis, metabolic disease, pancreas disorder, pure red-cell aplasia, skin disorder | Recruiting | Not stated | 2026-10-08 | NCT00027274 | |
| Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells | Bone Marrow Failure Disorders, Hemic and Lymphatic Diseases, Hemoglobinuria, Paroxysmal, anemia, aplastic anemia, bone marrow disorder, hematologic disorder, hemolytic anemia, myelodysplastic syndrome, myelodysplastic syndrome with excess blasts, myelodysplastic syndrome with single lineage dysplasia, pure red-cell aplasia | Recruiting | Phase 2 | 2026-09-22 | NCT01174108 | |
| T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias | Bone Marrow Failure Disorders, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Diamond-Blackfan anemia, Hemic and Lymphatic Diseases, anemia, aplastic anemia, beta thalassemia, beta-thalassemia major, bone marrow disorder, familial hemolytic anemia, hematologic disorder, hemolytic anemia, hereditary disease, inherited aplastic anemia, inherited hemoglobinopathy, pure red-cell aplasia, sickle cell disease, thalassemia | Recruiting | Phase 1 / Phase 2 | 2026-08-13 | NCT03653338 | |
| Assessing the Safety, Tolerability, and Efficacy of APR-2020 in Pediatric and Adolescent Subjects With RPS19 Deficient Diamond-Blackfan Anemia | Bone Marrow Failure Disorders, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Diamond-Blackfan anemia, Hemic and Lymphatic Diseases, anemia, aplastic anemia, bone marrow disorder, hematologic disorder, hereditary disease, inherited aplastic anemia, pure red-cell aplasia | Recruiting | Phase 1 | 2026-07-21 | NCT07476183 | |
| Familial Investigations of Childhood Cancer Predisposition | Abnormalities, Multiple, Adenomatous Polyps, Adnexal Diseases, Bone Diseases, Developmental, Bone Marrow Failure Disorders, Cardiovascular Abnormalities, Carney complex, Ciliopathies, Colorectal Neoplasms, Hereditary Nonpolyposis, Congenital Abnormalities, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cowden disease, Craniofacial Abnormalities, Cronkhite-Canada syndrome, Cysts, DICER1-related tumor predisposition, DNA repair disease, Diamond-Blackfan anemia, Digestive System Diseases, Digestive System Neoplasms, Disease Attributes, Ear Diseases, Eye Diseases, Hereditary, Fanconi anemia, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, GATA2 deficiency with susceptibility to MDS/AML, Genital Diseases, Hemic and Lymphatic Diseases, Hodgkins lymphoma, Hyperpigmentation, Infant, Newborn, Diseases, Jaw Cysts, Jaw Diseases, Li-Fraumeni syndrome, Lynch syndrome, Malformations of Cortical Development, Group I, Melanosis, Musculoskeletal Abnormalities, Myosarcoma, NF2-related schwannomatosis, Neoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Connective and Soft Tissue, Neoplasms, Germ Cell and Embryonal, Neoplasms, Multiple Primary, Neoplasms, Nerve Tissue, Neuroectodermal Tumors, Primitive, Neuroectodermal Tumors, Primitive, Peripheral, Noonan syndrome, Nutritional and Metabolic Diseases, Otorhinolaryngologic Neoplasms, PTEN hamartoma tumor syndrome, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Peutz-Jeghers syndrome, Rothmund-Thomson syndrome, Skin Abnormalities, Skin and Connective Tissue Diseases, Stomatognathic Diseases, Thoracic Neoplasms, Urogenital Diseases, Urogenital Neoplasms, X-linked disease, acoustic neuroma, acute leukemia, acute myeloid leukemia, adenocarcinoma, adenoma, adrenal cortex disorder, adrenal cortex neoplasm, adrenal gland cancer, adrenal gland disorder, anemia, angiomatosis, aplastic anemia, basal cell carcinoma, basal cell neoplasm, bone disorder, bone marrow disorder, breast disorder, breast neoplasm, carcinoma, cardiovascular disorder, central nervous system malformation, cerebral cortical dysplasia, choroid plexus carcinoma, classic familial adenomatous polyposis, colonic disorder, colorectal neoplasm, congenital heart disease, connective tissue disorder, connective tissue neoplasm, cranial nerve neoplasm, cranial nerve neuropathy, deafness-lymphedema-leukemia syndrome, digestive system disorder, digestive system neoplasm, disease susceptibility, dyskeratosis congenita, endocrine system disorder, epithelial neoplasm, eye disorder, familial rhabdoid tumor, female reproductive system disorder, female reproductive system neoplasm, gastrointestinal stromal tumor, gonadal disorder, hamartoma, heart cancer, heart disorder, hematologic disorder, hereditary Wilms tumor, hereditary breast ovarian cancer syndrome, hereditary disease, hereditary neoplastic syndrome, hereditary pheochromocytoma-paraganglioma, hereditary skin disorder, immune system disorder, immunoproliferative disorder, inherited aplastic anemia, inherited disease susceptibility, inherited neurodegenerative disorder, intestinal disorder, juvenile polyposis syndrome, lentigo, lymphatic system disorder, lymphoma, lymphoproliferative syndrome, malignant endocrine neoplasm, malignant pancreatic neoplasm, melanocytic neoplasm, melanoma, metabolic disease, mismatch repair cancer syndrome, mismatch repair cancer syndrome 1, multiple endocrine neoplasia, multiple endocrine neoplasia type 1, multiple endocrine neoplasia type 2A, musculoskeletal system disorder, myelodysplastic syndrome, myomatous neoplasm, myxoma, neoplasm, nerve sheath neoplasm, nervous system disorder, neuroblastoma, neurocutaneous syndrome, neurodegenerative disease, neuroendocrine neoplasm, neuroepithelial neoplasm, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neuroma, neuromuscular disease, nevoid basal cell carcinoma syndrome, non-Hodgkin lymphoma, ocular cancer, odontogenic cyst, otorhinolaryngologic disease, ovarian cancer, ovarian disorder, pancreas disorder, pancreatic neoplasm, paraganglioma, peripheral nervous system disorder, pheochromocytoma, pheochromocytoma-paraganglioma, primitive neuroectodermal tumor, pure red-cell aplasia, retinal cancer, retinal disorder, retinoblastoma, retrocochlear disease, rhabdomyosarcoma, sarcoma, schwannoma, skin disorder, skin neoplasm, skin pigmentation disorder, solitary bone cyst, tuberous sclerosis, vascular disorder, vestibulocochlear nerve disorder, von Hippel-Lindau disease | Recruiting | Not stated | 2026-06-17 | NCT03050268 | |
| Investigation of the Genetics of Hematologic Diseases | Bone Marrow Failure Disorders, Congenital Abnormalities, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DNA repair disease, Diamond-Blackfan anemia, Fanconi anemia, Hemic and Lymphatic Diseases, Kostmann syndrome, Nutritional and Metabolic Diseases, Skin Abnormalities, Skin and Connective Tissue Diseases, X-linked disease, anemia, aplastic anemia, blood coagulation disease, bone marrow disorder, dyskeratosis congenita, erythrocyte disorder, familial hemolytic anemia, hematologic disorder, hemolytic anemia, hereditary disease, hereditary skin disorder, inherited aplastic anemia, inherited hemoglobinopathy, leukocyte disorder, metabolic disease, myelodysplastic syndrome, pure red-cell aplasia, sickle cell disease, skin disorder | Recruiting | Not stated | 2026-06-09 | NCT02720679 | |
| Nonmyeloablative Stem Cell Transplant in Children With Sickle Cell Disease and a Major ABO-Incompatible Matched Sibling Donor | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, anemia, erythrocyte disorder, familial hemolytic anemia, hematologic disorder, hemolytic anemia, hereditary disease, inherited hemoglobinopathy, pure red-cell aplasia, sickle cell disease | Recruiting | Phase 2 | 2026-05-01 | NCT03214354 | |
| Mobilization of CD34+ Peripheral Blood Stem Cells in Patients With Diamond Blackfan Anemia Syndrome (DBAS) | Bone Marrow Failure Disorders, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Diamond-Blackfan anemia, Hemic and Lymphatic Diseases, anemia, aplastic anemia, bone marrow disorder, hematologic disorder, hereditary disease, inherited aplastic anemia, pure red-cell aplasia | Recruiting | Not stated | 2026-04-27 | NCT07186179 | |
| Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC | Blood Protein Disorders, Bone Diseases, Developmental, Bone Marrow Failure Disorders, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Chediak-Higashi syndrome, Chronic Disease, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Crohn disease, Cytopenia, DNA repair disease, Death, Death, Sudden, Diamond-Blackfan anemia, Digestive System Diseases, Disease Attributes, Eye Diseases, Hereditary, Gaucher disease, Hemic and Lymphatic Diseases, Hereditary Central Nervous System Demyelinating Diseases, Infant Death, Infant, Newborn, Diseases, Krabbe disease, Leukoencephalopathies, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lymphohistiocytosis, Hemophagocytic, Lysosomal Storage Diseases, Nervous System, Mannosidase Deficiency Diseases, Mucopolysaccharidosis I, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Peroxisomal Disorders, Primary Immunodeficiency Diseases, Skin and Connective Tissue Diseases, Sudden Infant Death, Sulfatidosis, Wiskott-Aldrich syndrome, X-linked disease, adrenal gland disorder, adrenocortical insufficiency, adrenoleukodystrophy, albinism, alpha-mannosidosis, anemia, aplastic anemia, arthritic joint disease, arthropathy, autoimmune disease, autoimmune lymphoproliferative syndrome, beta thalassemia, blood coagulation disease, bone disorder, bone marrow disorder, brain disorder, central nervous system disorder, chronic granulomatous disease, connective tissue disorder, cutaneous mucinosis, demyelinating disease, digestive system disorder, disease, dysgammaglobulinemia, endocrine system disorder, eye disorder, familial hemolytic anemia, gastroenteritis, hematologic disorder, hemolytic anemia, hemorrhagic disease, hereditary disease, histiocytosis, hyper-IgM syndrome, immune dysregulation-polyendocrinopathy-enteropathy-X-linked syndrome, immune system disorder, immunoproliferative disorder, inborn carbohydrate metabolic disorder, inborn error of immunity, inborn errors of metabolism, inflammatory bowel disease, inherited aplastic anemia, inherited blood coagulation disorder, inherited hemoglobinopathy, inherited neurodegenerative disorder, intellectual disability, intestinal disorder, leukocyte adhesion deficiency 1, leukocyte disorder, leukopenia, lymphatic system disorder, lymphopenia, lymphoproliferative syndrome, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, metachromatic leukodystrophy, mucopolysaccharidosis, musculoskeletal system disorder, nervous system disorder, non-Langerhans cell histiocytosis, osteochondrodysplasia, osteopetrosis, osteosclerosis, phagocyte bactericidal dysfunction, pure red-cell aplasia, rheumatic disorder, rheumatoid arthritis, severe combined immunodeficiency, sickle cell disease, sphingolipidosis, syndromic disease, thalassemia | Recruiting | Not stated | 2026-01-13 | NCT04528355 | |
| Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT | Bone Diseases, Developmental, Bone Marrow Failure Disorders, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Chediak-Higashi syndrome, Chronic Disease, Congenital Abnormalities, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Crohn disease, Cytopenia, DNA repair disease, Death, Death, Sudden, Diamond-Blackfan anemia, Digestive System Diseases, Disease Attributes, Eye Diseases, Hereditary, Gaucher disease, Hemic and Lymphatic Diseases, Hereditary Central Nervous System Demyelinating Diseases, Infant Death, Infant, Newborn, Diseases, Krabbe disease, Leukoencephalopathies, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lymphohistiocytosis, Hemophagocytic, Lysosomal Storage Diseases, Nervous System, Mannosidase Deficiency Diseases, Mucopolysaccharidosis I, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Peroxisomal Disorders, Primary Immunodeficiency Diseases, Skin Abnormalities, Skin and Connective Tissue Diseases, Sudden Infant Death, Sulfatidosis, Wiskott-Aldrich syndrome, X-linked disease, adrenal gland disorder, adrenocortical insufficiency, adrenoleukodystrophy, albinism, alpha-mannosidosis, anemia, aplastic anemia, arthritic joint disease, arthropathy, autoimmune disease, autoimmune lymphoproliferative syndrome, beta thalassemia, blood coagulation disease, bone disorder, bone marrow disorder, brain disorder, central nervous system disorder, chronic granulomatous disease, connective tissue disorder, cutaneous mucinosis, demyelinating disease, digestive system disorder, disease, dyskeratosis congenita, endocrine system disorder, eye disorder, familial hemolytic anemia, gastroenteritis, hematologic disorder, hemolytic anemia, hemorrhagic disease, hereditary disease, hereditary skin disorder, histiocytosis, immune dysregulation-polyendocrinopathy-enteropathy-X-linked syndrome, immune system disorder, immunoproliferative disorder, inborn carbohydrate metabolic disorder, inborn error of immunity, inborn errors of metabolism, inflammatory bowel disease, inherited aplastic anemia, inherited blood coagulation disorder, inherited hemoglobinopathy, inherited neurodegenerative disorder, intellectual disability, intestinal disorder, juvenile idiopathic arthritis, leukocyte adhesion deficiency 1, leukocyte disorder, leukoencephalopathy, diffuse hereditary, with spheroids 1, leukopenia, lymphatic system disorder, lymphopenia, lymphoproliferative syndrome, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, metachromatic leukodystrophy, mucopolysaccharidosis, mucopolysaccharidosis type 2, musculoskeletal system disorder, nervous system disorder, non-Langerhans cell histiocytosis, osteochondrodysplasia, osteopetrosis, osteosclerosis, phagocyte bactericidal dysfunction, pure red-cell aplasia, rheumatic disorder, severe combined immunodeficiency, sickle cell disease, skin disorder, sphingolipidosis, syndromic disease, thalassemia | Recruiting | Phase 2 | 2025-12-15 | NCT01962415 | |
| CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation | AIDS, Blood-Borne Infections, Bone Marrow Failure Disorders, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Diamond-Blackfan anemia, Digestive System Diseases, Familial Hemophagocytic Lymphocytosis, Genital Diseases, HIV infectious disease, Hemic and Lymphatic Diseases, Histiocytosis, Langerhans-Cell, Infections, Kostmann syndrome, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lymphohistiocytosis, Hemophagocytic, Nutritional and Metabolic Diseases, Primary Immunodeficiency Diseases, RNA Virus Infections, Shwachman-Diamond syndrome, Slow Virus Diseases, Urogenital Diseases, anemia, aplastic anemia, bone marrow disorder, exocrine pancreatic insufficiency, familial hemolytic anemia, hematologic disorder, hemolytic anemia, hereditary disease, histiocytosis, immune system disorder, immunoproliferative disorder, inborn error of immunity, infectious disease, inherited aplastic anemia, inherited hemoglobinopathy, interstitial lung disease, lentivirus infection, lipomatosis, lung disorder, lymphatic system disorder, lymphoproliferative syndrome, macrophage activation syndrome, metabolic disease, non-Langerhans cell histiocytosis, pancreas disorder, pure red-cell aplasia, respiratory system disorder, sexually transmitted disease, sickle cell disease, sickle cell-beta-thalassemia disease syndrome, uterine carcinosarcoma, viral infectious disease, viral sexually transmitted disease | Active, not recruiting | Phase 1 / Phase 2 | 2025-08-14 | NCT01966367 | |
| AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion | Bone Marrow Failure Disorders, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Diamond-Blackfan anemia, Hemic and Lymphatic Diseases, Hodgkins lymphoma, Kostmann syndrome, Neoplasms by Histologic Type, acute leukemia, anemia, aplastic anemia, beta thalassemia, bone marrow disorder, familial hemolytic anemia, hematologic disorder, hemolytic anemia, hereditary disease, immune system disorder, immunoproliferative disorder, inherited aplastic anemia, inherited hemoglobinopathy, lymphatic system disorder, lymphoma, lymphoproliferative syndrome, neoplasm, non-Hodgkin lymphoma, pure red-cell aplasia, sickle cell disease, thalassemia | Recruiting | Phase 2 | 2025-08-08 | NCT04099966 | |
| National Registry of Rare Kidney Diseases | 46 XY differences of sex development, AH amyloidosis, Abnormalities, Multiple, Alport syndrome, Amino Acid Transport Disorders, Inborn, Arterial Occlusive Diseases, Bartter syndrome, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Cerebral Small Vessel Diseases, Ciliopathies, Collagen Diseases, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cytopenia, Dent disease, Denys-Drash syndrome, EAST syndrome, Fabry disease, Fanconi renotubular syndrome 1, Fanconi renotubular syndrome 2, Fanconi renotubular syndrome 3, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Fibrosis, Gitelman syndrome, Glycosuria, Hemic and Lymphatic Diseases, Hyperoxaluria, IgA glomerulonephritis, IgG4-related retroperitoneal fibrosis, Immunoglobulin Light-chain Amyloidosis, Liddle syndrome, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Male Urogenital Diseases, Malformations of Cortical Development, Group I, Neoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Multiple Primary, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Polycystic Kidney, Autosomal Recessive, Skin and Connective Tissue Diseases, Urination Disorders, Urogenital Abnormalities, Urogenital Diseases, Urogenital Neoplasms, Wilms tumor, X-linked disease, adenine phosphoribosyltransferase deficiency, adrenal gland disorder, adrenal gland hyperfunction, amyloidosis, anemia, arthropathy, atypical hemolytic-uremic syndrome, autoimmune disease, autosomal dominant polycystic kidney disease, autosomal recessive proximal renal tubular acidosis, benign urinary system neoplasm, blood platelet disease, brain disorder, calcinosis, calciphylaxis, calcium metabolic disease, cardiovascular disorder, central nervous system disorder, central nervous system malformation, cerebral cortical dysplasia, cerebrovascular disorder, complement 3 glomerulopathy, connective tissue disorder, cystic kidney disease, cystinosis, cystinuria, diabetes insipidus, disorder of sexual differentiation, endocrine system disorder, familial primary hypomagnesemia with hypocalcuria, familial renal glucosuria, fibromuscular dysplasia, focal segmental glomerulosclerosis, glomerulonephritis, glucose metabolism disease, gonadal disorder, hamartoma, hematologic disorder, hematuria, benign familial, hemolytic anemia, hemolytic-uremic syndrome, hereditary disease, hereditary hypophosphatemic rickets with hypercalciuria, hereditary neoplastic syndrome, hereditary nephritis, hereditary renal hypouricemia, hyperaldosteronism, immune system disorder, immunoproliferative disorder, inborn carbohydrate metabolic disorder, inborn errors of metabolism, inherited neurodegenerative disorder, intestinal hypomagnesemia 1, kidney cancer, kidney disorder, lymphoproliferative syndrome, lysosomal lipid storage disorder, lysosomal storage disease, membranoproliferative glomerulonephritis, membranous glomerulonephritis, metabolic disease, minimal change disease, mixed neoplasm, monoclonal gammopathy, musculoskeletal system disorder, nail disorder, nail-patella syndrome, neoplasm, nephritis, nephrogenic diabetes insipidus, nephrogenic syndrome of inappropriate antidiuresis, nephronophthisis, nephronophthisis 1, nephrosis, nephrotic syndrome, nephrotic syndrome of childhood - steroid sensitive, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, oculocerebrorenal syndrome, plasma cell neoplasm, polycystic kidney disease, primary hyperoxaluria, proteostasis deficiencies, pseudohypoaldosteronism, pseudohypoaldosteronism, type IB1, autosomal recessive, pure red-cell aplasia, renal aminoaciduria, renal cysts and diabetes syndrome, renal hypomagnesemia 3, renal tubular acidosis, distal, 3, with or without sensorineural hearing loss, renal tubular transport disease, skin disorder, sphingolipidosis, steroid-resistant nephrotic syndrome, thrombocytopenia, thrombotic microangiopathy, tuberous sclerosis, uremia, urinary system disorder, vascular disorder, vasculitis | Recruiting | Not stated | 2023-10-04 | NCT06065852 | |
| French National Registry of Bone Marrow Failures | Bone Marrow Failure Disorders, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DNA repair disease, Diamond-Blackfan anemia, Fanconi anemia, Hemic and Lymphatic Diseases, Nutritional and Metabolic Diseases, anemia, aplastic anemia, bone marrow disorder, hematologic disorder, hereditary disease, inherited aplastic anemia, metabolic disease, pure red-cell aplasia | Recruiting | Not stated | 2021-03-04 | NCT04781790 |